Friedreich's Ataxia Insights

This week's must-know community updates, latest research & events

March 13, 2025 • Estimated Reading Time: 1 minute

New This Week

Exciting news! There is now a new section featuring clinical trials that are actively recruiting patients. This highly requested addition gives you direct access to cutting-edge research opportunities that could shape the future of treatment.

Stay informed about potentially life-changing studies and let us know what you think by replying to this email!

Latest Research

The study by Burt et al. (2025) aimed to validate the Patient-Reported Outcome Measure of Ataxia (PROM-Ataxia) over time. They analyzed data from 176 patients with various types of spinocerebellar ataxia, including Friedreich's Ataxia, over a year. The PROM-Ataxia showed high internal consistency and was sensitive to changes in disease severity, correlating well with other clinical outcome assessments.

The progression of symptoms followed a sigmoidal pattern, with the most significant changes observed in patients with moderate severity. The study found that mental aspects deteriorated quickest in mild cases, physical aspects in moderate cases, and daily living activities in severe cases. Burt et al. concluded that PROM-Ataxia is a sensitive tool for capturing the evolution of patient experiences with ataxia over time, outperforming other clinical outcome assessments in detecting changes.

Clinical Trials

This is a list of upcoming or ongoing clinical trials that are actively recruiting and have been listed or updated in the last two weeks:

A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it for the First Time

View the full list of clinical trials →

Community News

National Ataxia FoundationMar 12, 2025

Did you know? The cerebellum is the part of the brain responsible for movement coordination in the body. Ataxia symptoms occur when the cerebellum is not functioning properly. Cerebellar dysfunction can be caused genetically like in the SCAs or by physical injury. #AtaxiaAwareness

As part of @danafoundation #BrainAwarenessWeek, we'll be sharing facts about Ataxia all week long! Share, like, and comment on our posts to help create awareness for #Ataxia!

Cure FAMar 10, 2025

Today, March 10, 2025, Design Therapeutics announced progress across its lead GeneTAC® programs including DT-216P2, a candidate being developed for the treatment of FA. A Phase 1 dosing study in healthy volunteers has been initiated in Australia to evaluate the safety and pharmacokinetics (PK) of single ascending doses of DT-216P2 via multiple routes of administration (intravenous infusion, subcutaneous infusion and subcutaneous injection). A Phase 1/2 multiple ascending dose (MAD) clinical trial to assess safety, PK and pharmacodynamics (PD) in FA patients is anticipated to begin in mid-2025.

Click here to read the full press release: https://ow.ly/cmjr50VeN6w

Click here to read more about the DT-216P2 program: https://ow.ly/GO2l50VeN6v

Ataxia UKMar 12, 2025

A new study is recruiting participants, which aims to understand how Friedreich’s Ataxia (FA) impacts your daily life and overall health. Taking part involves completing a 30-minute survey. If you are aged 12 or over and have FA, or provide care for someone with FA, find out if you can take part here: https://www.ataxia.org.uk/wp-content/uploads/2025/01/FA-study-invitation-for-social-media.pdf.

#AtaxiaUK#Research#Takepart